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About the Regulatory Profession

The regulatory function is vital in making safe and effective healthcare products available worldwide. Individuals who ensure regulatory compliance and prepare submissions, as well as those whose main job function is clinical affairs or quality assurance are all considered regulatory professionals.

Regulatory Code of Ethics

One of our most valuable contributions to the profession is the Regulatory Code of Ethics. The Code of Ethics provides regulatory professionals with core values that hold them to the highest standards of professional conduct.

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Regulatory Convergence

Join the brightest minds in regulatory at the annual Regulatory Convergence. See the global regulatory community in action. Intensive workshops. Topical sessions. Meet ups with regulators. This is where it all comes together.

Republican Senators Lobby FDA Ahead of Decision on Sarepta’s DMD Drug

The contentious debate over whether the US Food and Drug Administration (FDA) will approve or deny Sarepta’s Duchenne Muscular Dystrophy (DMD) drug eteplirsen will come to an end either on or before Thursday of this week.

But before that decision is made, two Republican senators, Ron Johnson (WI), Chairman of the Committee on Homeland Security and Governmental Affairs, and Dan Coats (IN), sent a letter to FDA Commissioner Robert Califf last Friday to “express disappointment in [an advisory committee of outside experts’] vote against approval of the new drug.”

Part of the disappointment, Coats and Johnson said, was tied to the questions posed to the advisory committee, which “may be framed in such a way that could make it more difficult than necessary for members to vote favorably for an application.”

The committee, which voted 7-3 against approval in April, raised serious concerns about the way in which the eteplirsen 12-subject trial was conducted, though parents of the young boys with DMD who were enrolled in the trial lobbied hard at the meeting for the drug’s approval.

CDER Director Janet Woodcock, who rarely attends advisory committee hearings, also made clear that FDA’s final decision will not be a straightforward one (the agency is not required to follow the opinion of its advisory committee, though it often does) as FDA and Sarepta differed widely in their analyses of whether the treatment was effective, though both sides agreed that the treatment is safe.

Johnson and Coats, however, called on FDA to employ certain “flexibilities” in allowing potential treatments for life-threatening diseases, such as eteplirsen (though as Sarepta made clear at the meeting, eteplirsen is meant to help the young boys with DMD to remain mobile, it’s not a cure for DMD).

“Congress has multiple times attempted to provide the FDA with the tools and necessary authority to speed access to drugs and therapies where the costs of delay and certainty of efficacy are far outweighed by its potential benefit,” the senators wrote to Califf. “This is especially true in small disease populations where post-approval confirmatory trials can substitute for large pre-approval randomized efficacy trials that are extremely difficult or impossible to conduct.”

But as Eric Bastings, FDA deputy director of FDA’s Office of Drug Evaluation’s Division of Neurology Projects, noted in the advisory committee meeting: “Accelerated approval cannot be used to compensate for weak or insufficient data.”